FDA
2nd February 2016

FDA Denies Approval of Drisapersen for the Treatment of DMD

The FDA has recently issued a Complete Response Letter to BioMarin Pharmaceuticals, indicating that there was insufficient evidence of effectiveness of drisapersen in the treatment of Duchenne Muscular Dystrophy (DMD) to support approval.

FDA Denies Approval of Drisapersen for the Treatment of DMD

The FDA has recently issued a Complete Response Letter to BioMarin Pharmaceuticals, indicating that there was insufficient evidence of effectiveness of drisapersen in the treatment of Duchenne Muscular Dystrophy (DMD) to support approval.

DMD, a rare genetic disorder resulting in progressive muscle degeneration, is the result of a genetic error in messenger RNA splicing, in which an exon is “skipped.” Drisapersen is a chemically modified antisense oligonucleotide (20-mer) that specifically binds to exon 51 of the human dystrophin pre-mRNA. The drug is specifically designed to cause the skipping of exon 51, which causes the generation of a shortened dystrophin.

In November of 2015, the Peripheral and Central Nervous System (PCNS) Drugs Advisory Committee convened to discuss the efficacy and safety of drisapersen for the treatment of DMD.

While the committee acknowledged the difficulty in studying this rare disease, as well as the need for therapies, the panel concluded that the data presented for the three clinical trials did not provide sufficient evidence of the drug’s efficacy or benefit-risk profile. In a final vote, the majority of the committee (10 members) agreed that the data presented about dystrophin production had little effect on the interpretation of the clinical results.

Panel member, Michelle M. Mielke, PhD, associate professor, Division of Epidemiology, Department of Health Sciences Research, Mayo Clinic, Rochester, Minnesota, said that drisapersen may be beneficial for “particular individuals with certain characteristics or if there was a loading dose.”

Valerie Cwik, MD, Muscular Dystrophy Association Vice President and Chief Medical and Scientific Officer commented on the FDA’s verdict, stating that, “our families urgently need safe and effective treatments, and although this is not the outcome so many had hoped for, we know that the FDA exercised its due diligence in considering drisapersen.”

In the U.S., there are currently no FDA-approved therapies for the condition; however, two other drugs for the treatment of DMD will be considered by the FDA in 2016. According to BioMarin, ongoing drisapersen extension studies and clinical trials will continue. Additionally, patients currently receiving drisapersen and the other products will remain on therapy.

ISS has over a decade of experience in developing regulatory strategies including support for FDA Advisory Committee meetings. We are involved in more FDA AdComms per year than even the largest pharmaceutical companies. For more information on how ISS can help you prepare for your next meeting, contact info@innovativescience.net

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